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Axatilimab With or Without Azacitidine for the Treatment of Patients With Advanced Phase Myeloproliferative Neoplasms, Myeloproliferative Neoplasm/Myelodysplastic Syndrome Overlap or High Risk Chronic Myelomonocytic Leukemia

ConditionsAtypical Chronic Myeloid LeukemiaChronic Myelomonocytic LeukemiaMyelodysplastic/Myeloproliferative NeoplasmRecurrent Myelodysplastic/Myeloproliferative NeoplasmRecurrent Myeloproliferative NeoplasmRefractory Chronic Myelomonocytic LeukemiaRefractory Myelodysplastic/Myeloproliferative NeoplasmRefractory Myeloproliferative Neoplasm
Trial statusRecruiting
Trial phasePhase 1, Phase 2
Trial typeInterventional
Biological sexAll
Age18+
SponsorUma Borate

About this trial

This phase Ib/II trial tests the best dose of axatilimab and effectiveness of axatilimab with or without azacitidine for the treatment of patients with advanced phase myeloproliferative neoplasms (MPN), myeloproliferative neoplasm/myelodysplastic syndrome (MPN/MDS) overlap or high risk chronic myelomonocytic leukemia (CMML). Axatilimab is an antibody that is cloned from a single white blood cell that is known to be able to recognize cancer cells and block a protein on the surface of the white blood cells that may be involved in cancer cell growth. By blocking the proteins, this may slow or halt the growth of the cancer. Azacitidine is in a class of medications called antimetabolites. It works by stopping or slowing the growth of cancer cells. Giving axatilimab with or without azacitidine may be safe and effective in treating patients with advanced phase MPN, MPN/MDS overlap or high risk CMML.

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

Signed informed consent must be obtained prior to participation in the study

Age ≥ 18 years at the date of signing the informed consent form (ICF)

Chronic myelomonocytic leukemia (CMML), classified as intermediate-2, OR high-risk per the CMML Specific Prognostic Scoring System (CPSS) Molecular Model

Atypical chronic myelocytic leukemia (aCML)

Disqualifiers

Diagnosis of acute myeloid leukemia (AML) including acute promyelocytic leukemia and extra-medullary AML based on WHO 2016 classification (Arber et al 2016)

Patients who are candidates for myeloablative or intensive chemotherapy treatment or who do not provide consent for this treatment

History of organ transplant or allogenic hematopoietic stem cell transplant

Participants with history of adequately treated malignancy for which no anticancer systemic therapy (namely chemotherapy, radiotherapy or surgery) is ongoing or required during the course of the study.

Trial design

Design model

Crossover

Treatments tested in this trial

  • Axatilimab

    Biological/Vaccine

    Given IV

  • Azacitidine

    Drug

    Given IV or SC

  • Biospecimen Collection

    Procedure/Surgery

    Undergo blood sample collection

  • Bone Marrow Aspiration and Biopsy

    Procedure/Surgery

    Undergo bone marrow biopsy and aspiration

  • Survey Administration

    Other intervention

    Ancillary study

Treatment groups

49 Participants
are divided into 2 treatment groups
Group A: Phase I (Axatilimab)Experimental treatment 3 interventions
Group B: Phase II (Axatilimab and azacitidine)Experimental treatment 5 interventions

Trial outcomes

Primary outcomes

1

Incidence of dose limiting toxicities

Includes hematologic and non-hematologic toxicities

Time frame
Up to 42 days after the first dose of study medication
2

Overall response rate

The number of participants whose best response (according to the 2006 International Working Group response criteria) over the efficacy analysis period is a complete response or partial response will be tallied to estimate the overall response rate and provide a 95% exact confidence interval, according to the Clopper-Pearson method.

Time frame
Up to 5 years

Secondary outcomes

1

Incidence of adverse events

Adverse events will be tabulated by toxicity grade and relationship with study medication and assessed by Common Terminology Criteria for Adverse Events version 5.0.

Time frame
Up to 30 days after end of treatment
2

Quality of life measured by Modified Myeloproliferative Neoplasm Symptom Assessment Form (MPNSAF)

Will be assessed in patients receiving axatilimab + azacitidine (AZA) and will be determined using a Modified Myeloproliferative Neoplasm Symptom Assessment Form (MPNSAF) score. The MPNSAF score has a range from 0 to 100 with 100 representing the highest level of symptom severity. Descriptive statistics, means and standard deviations or medians and ranges will be applied to summarize the MPNSAF score.

Time frame
Up to 5 years
3

Number of platelet transfusions

Will be assessed in patients receiving axatilimab + azacitidine and will be determined by descriptive statistics.

Time frame
Up to 5 years
4

Number of packed red blood cell transfusions

Will be assessed in patients receiving axatilimab + azacitidine and will be determined by descriptive statistics.

Time frame
Up to 5 years

Other outcomes

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

Uma Borate

Lead sponsor

Ohio State University Comprehensive Cancer Center

Sponsor institution

Incyte Corporation

Collaborator

Source ClinicalTrials.gov
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